Novartis is celebrating a significant achievement as its investigational oral factor B inhibitor reaches a crucial milestone in a phase 3 clinical trial for a kidney disorder. The positive outcome has prompted the pharmaceutical giant to prepare for a fast-track FDA approval request in the coming year.
The noteworthy data emerges from Novartis’ APPLAUSE-IgAN study, a 9-month endeavor. While the full results are anticipated in 2025, this double-blind, placebo-controlled trial primarily aims to assess the safety and efficacy of oral iptacopan, administered twice daily, among 470 adults afflicted with IgA nephropathy (IgAN), a chronic kidney ailment also known as Berger’s disease.
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Iptacopan has successfully met the intermediary primary endpoint in the trial, displaying statistically significant superiority over placebo in terms of reducing proteinuria, as reported by Novartis. However, specific figures have yet to be disclosed. The study will continue to evaluate the pill’s potential to decelerate IgAN progression by monitoring the estimated glomerular filtration rate slope over a two-year period.
Financial analyst group ODDO BHF lauds this development as a “faultless” performance by iptacopan, characterizing it as a home run for Novartis. Beyond the prospect of becoming the inaugural oral monotherapy for IgAN patients, iptacopan also holds the potential to be the first targeted treatment for IgAN that effectively halts or prevents progression to kidney failure by activating the complement system, according to Novartis.
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Notably, APPLAUSE-IgAN has demonstrated a safety profile consistent with data previously reported in two phase 3 trials for paroxysmal nocturnal hemoglobinuria (PNH). Capitalizing on this success, Novartis has already submitted iptacopan for regulatory approval in the rare disease indication, with a response expected in 2024. If approved for PNH, Novartis is poised to challenge AstraZeneca’s rare disease unit Alexion with its complement inhibitors Soliris and Ultomiris. In a previous head-to-head PNH trial, iptacopan outperformed Soliris.
ODDO BHF analysts assert that iptacopan holds the potential of being a “pipeline in a pill.” This versatile medication is also under development for various other renal and hematological conditions currently underserved by available treatments. ODDO BHF anticipates an initial iptacopan launch in 2024, with peak sales potentially exceeding $3 billion across all indications. This promising development signifies a major stride in the quest for improved treatments in the field of kidney disorders.
